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Erişkin herediter anjiödem hastalarında danazol ile yapılan uzun dönem profilaksi tedavisine uyumun değerlendirilmesi: Gerçek bir yaşam çalışması

Year 2019, Volume: 32 Issue: 1, 7 - 13, 29.01.2019
https://doi.org/10.5472/marumj.518719

Abstract

Amaç: Herediter anjioödem (HAÖ) hastalarında profilaktik
tedaviye uyumu ve bunları etkileyen olası faktörleri araştırmaktır.
Hastalar ve Yöntemler: Altmış HAÖ hastasının tıbbi kayıtları
değerlendirildi ve demografik ve hastalıkları ile ilgili klinik
özellikler ve kullandıkları ilaçlar ile ilgili soruları içeren anketi
yanıtlamaları istendi. Hastalık ciddiyeti, semptomların başlangıç
yaşı, klinik bulguları ve uzun dönem profilaksi ihtiyacına göre
belirlendi.
Bulgular: Hastaların %65’i kadın, yaş ortalaması 38,07±12,38,
%93,3’ü tip 1 HAÖ, %58,3’ünün hastalığı ciddi idi ve %71,7’si
danazol ile profilaktik tedavi görüyordu. Ondört hasta proflaktik
tedavisini düzenli kullanmıyordu ve kullanmama nedenleri yan
etki gelişmesinden korkmak (n=11) ve ilacı almayı unutmak idi
(n=4). Ailede tek vaka olanların, HAÖ’li akrabası az olanların ve
ailesinde bu nedenle hayatlarını kaybeden akrabası olmayanların
profilaktik tedaviye daha uyumlu olduğu görüldü (P=0,008;
P=0,018; P=0,028).
Sonuç: Hastalarımızın büyük çoğunluğu proflaktik tedaviyi
düzenli kullanmakta ve bu tedaviden fayda görmektedir.
Uyumsuzluğun primer nedeni yan etki gelişimi korkusudur.

References

  • 1. Cicardi M, Aberer W, Banerji A, et al. Classification, diagnosis and approach to treatment for angioedema: consesnsus report from the Hereditary Angioedema International Working Group. Allergy 2014;69:602-16. doi: 10.1111/all.12380
  • 2. Zuraw BL. Hereditary angioedema. N Engl J Med 2008;359:1027-36. doi: 10.1056/NEJMcp0803977.
  • 3. Gompels MM, Lock RJ, Abinun M, et al. C1 inhibitor deficiency: consensus document. Clin Exp Immunol 2005;139:379-94.
  • 4. Zilberberg M, Jacobsen T, Tillotson G. The burden of hospitalizations and emergency department visits with hereditary angioedema and angioedema in the United States, 2007. Allergy Asthma Proc 2010;31:511-9. doi: 10.2500/ aap.2010.31.3403.
  • 5. Roche O, Blanch A, Caballero T, Sastre N, Callejo D, Lopez- Trascasa M. Hereditary angioedema due to C1 inhibitor deficiency: patient registry and approach to the prevelance in Spain. Ann Allergy Asthma Immunol 2005;94:498-503.
  • 6. Bygum A. Hereditary angioedema in Denmark: a nationwide survey. Br J Dermatol 2009;161:1153-8. doi: 10.1111/j.1365- 2133.2009.09366.x.
  • 7. Craig T, Aygören-Pürsün E, Bork K, et al. WAO Guideline for the management of hereditary angioedema. World Allergy Organ J 2012;5:182-9. doi: 10.1097/ WOX.0b013e318279affa.
  • 8. Caballero T, Baeza ML, Cabanas R, et al. Consensus statement on the diagnosis, management and the treatment of angioedema mediated by bradikynin. Part II. Treatment, follow up, and special situations. J Investig Allergol Clin Immunol 2011;21:422-41.
  • 9. Costantino G, Casazza G, Bossi I, Duca P, Cicardi M. Longterm prophylaxis in hereditary angio-edema: a systematic review. BMJ Open 2012;2:e000524. doi: 10.1136/ bmjopen-2011-000524.
  • 10. Wilson DA, Bork K, Shea EP, Rentz AM, Blaustein MB, Pullman WE. Economic costs associated with acute attacks and long term management of hereditary angioedema. Ann Allergy Asthma Immunol 2010;104:314-20. doi: 10.1016/j. anai.2010.01.024.
  • 11. Lumry WR, Castaldo AJ, Vernon MK, Blaustein MB, Wilson DA, Horn PT. The humanistic burden of hereditary angioedem: Impact on health-related quality of life, productivity, and depression. Allergy Asthma Proc 2010;31:407-14. doi:10.2500/aap.2010.31.3394.
  • 12. Nordenfelt P, Dawson S, Wahlgren CF, Lindfors A, Malbris L, Björkander J. Quantifying the burden of disease health state in patients with hereditary angiopedema in Sweeden. Allergy Asthma Proc 2014;35:185-90. doi: 10.2500/ aap.2014.35.3738.
  • 13. Cabellero T, Aygören-Pürsün E, Bygum A, et al. The humanistic burden of hereditary angioedema: results from the Burden of Illness Study in Europe. Allergy Asthma Proc 2014;35:47-53. doi: 10.2500/aap.2013.34.3685.
  • 14. Hughes DA, Bagust A, Haycox A, Walley T. Accounting for noncompliance in pharmacoeconomic evaluations. Pharmacoeconomics 2001;19:1185-97.
  • 15. Hudhes DA. Economic impact of poor compliance with pharmaceuticals. Expert Rev Pharmacoecon Outcomes Res 2002;2:327-35. doi: 10.1586/14737167.2.4.327
  • 16. Bygum A, Fagerberg CR, Ponard D, Monnier N, Lunardi J, Drouet C. Mutational spectrum and phenotypesin Danish families with hereditary angioedema because of C1 inhibitor deficiency. Allergy 2011;66:76-84. doi: 10.1111/j.1398- 9995.2010.02456.x
  • 17. Squeglia V, Barbarino A, Bova M, et al. High attack frequency in patients with angioedema due to C1-inhibitor deficiency is a major determinant in switching to home therapy: a real-life observational study. Orphanet J Rare Dis 2016; 11:133.doi: 10.1186/s13023.016.0518-8.
  • 18. Nordenfelt P, Nilsson M, Björkander J, Mallbris L, Lindfors A, Whalgren CF. Hereditary angioedema in Swedish adults: report from the national cohort. Acta Derm Venerol 2016;96:540-5. doi: 10.2340/00015.555.2274.
  • 19. Gomez-Traseira C, Perez-Fernandez E, Lopez-Serrano MC, et al. Clinical pattern and acute and long-term management of hereditary angioedema due to C1-esterase inhibitor deficiency. J Investig Allergol Clin Immunol 2015;25:358- 64.
  • 20. Zanichelli A, Vacchini R, Badini M, Penna V, Cicardi M. Standard care impact on angioedema because of hereditary C1 inhibitor deficiency: a 21-month prospective study in a cohort of 103 patients. Allergy 2011;66:192-6. doi: 10.1111/j.1398-9995.2010.02433.x.
  • 21. Ren HL, Zhang HY. Clinical features of hereditary angioedema: analysis of 133 cases. Zhonghua Yi Xue Za Zhi 2007;87:2772-6.
  • 22. Gelfand JA, Sherins RJ, Alling DW, Frank MM. Treatment of hereditary angioedema with Danazol. Reversal of clinical and biochemical abnormalities. N Engl J Med 1976;295:1444-8. doi: 10.1056/NEJM197.612.232952602.
  • 23. Bork K, Bygum A, Hardt J. Benefits and risks of Danazol in hereditary angioedema: a long term survey of 118 patients. Ann Allergy Asthma Immunol 2008;100:153-61. doi: 10.1016/S1081-1206(10)60424-3.
  • 24. Riedl MA. Critical appraisal of androgen use in hereditary angioedema: a systematic review. Ann Allergy Asthma Immunol 2015;114:281-8. doi: 10.1016/j.anai.2015.01.003.
  • 25. Cicardi M, Suffritti C, Perego F, Caccia S. Novelties in the diagnosis and treatment of angioedema. J Investig Allergol Clin Immunol 2016;26:212-1. doi: 10.18176/jiaci.0087
  • 26. Farkas H, Veszeli N, Csuka D, et al. Management of pregnancies in a hereditary angioedema patients after treatment with attenuated androgens since childhood. J Obstet Gynaecol 2015;35:89-90. doi: 10.3109/01443.615.2014.925860.
  • 27. Zuraw BL, Busse PJ, White M, et al. Nanofiltered C1 inhibitor concentrate for treatment of hereditary angioedema. N Engl J Med 2010;363:513-22. doi: 10.1056/NEJMoa0805538.
  • 28. Craig T, Shapiro R, Vegh A, et al. Efficacy and safety of an intravenous C1-inhibitor concentrate for long-term prophylaxis in hereditary angioedema. Allergy Rhinol (Providence). 2017:1:8:13-19. doi: 10.2500/ar.2017.8.0192
  • 29. Ucar R, Arslan S, Baran M, Caliskaner AZ. Difficulties encountered in the emergency department by patients with hereditary angioedema experiencing acute attacks. Allergy Asthma Proc 2016;37:72-5. doi: 10.2500/aap.2016.37.3905.
  • 30. Kesim B, Uyguner ZO, Gelincik A, et al. The Turkish hereditary angioedema pilot study (TURHAPS): the first Turkish series of hereditary angioedema. Int Arch Allergy Immunol 2011;156:443-50. doi:10.1159/000323915.
  • 31. Zanichelli A, MagerlM, Longhurst H, Fabien V, Maurer M. Hereditary angioedema with C1 inhibitor deficiency: delay indiagnosis in Europe. Allergy Asthma Clin Immunol 2013;9:29. doi: 10.1186/1710-1492-9-29.
  • 32. Frank MM, Gelfand JA, Atkinson JP. Herediatry angioedema: the clinical syndrome and its management. Ann Intern Med 1976;84:580-93.
  • 33. Agostoni A, Aygören-Pürsün E, Binkley KE, et al. Hereditary and acquired angioedema: problems and progress: proceedings of the third C1 esterase inhibitör deficiency workshop and beyond. J Allergy Clin Immunol 2004;114(3 Suppl):S51-131.
  • 34. Agostoni A. Cicardi M. Hereditary and acquired C1-inhibitor deficiency: biological and clinical characteristics in 235 patients. Medicine (Baltimore) 1992;71:206-15.
  • 35. Bork K, Meng G, Staubach P, Hardt J. Hereditary angioedema: new findings concerning symptoms, affected organs, and course. Am J Med 2006;119:267-74.

Evaluating adherence to long-term prophylaxis treatment with danazol in adult hereditary angioedema patients: A real life study

Year 2019, Volume: 32 Issue: 1, 7 - 13, 29.01.2019
https://doi.org/10.5472/marumj.518719

Abstract

Objective: To investigate the adherence to the prophylactic
treatment in hereditary angioedema (HAE) patients as well as the
potential factors which may affect this situation.
Patients and Methods: In addition to evaluation of their
medical records, sixty HAE patients were asked to complete a
questionnaire including inquiries about demographic and clinical
features of their disease and medications used. Disease severity
was determined depending on their age of onset of symptoms,
clinical manifestations, and need of long-term prophylaxis.
Results: Sixty-five percent of the patients were female, the
mean age was 38.07±12.38 years, 93.3% were type 1 HAE, 58.3%
had a severe form of the disease, and 71.7% were under prophylaxis
with danazol. Fourteen patients were not using danazol regularly
due to the fear of side effects (n=11) and forgetfulness to take the
medication (n=4). It was observed that the patients who were the
only cases in their families, those having few relatives with HAE
and having had no excitus due to HAE in their families, were more
adherent to prophylactic treatment (P=0.008; P=0.018; P=0.028).
Conclusion: The majority of patients were effectively under
long-term prophylaxis and the majority adhered to this treatment.
The primary cause of non-adherence was fear of side effects.

References

  • 1. Cicardi M, Aberer W, Banerji A, et al. Classification, diagnosis and approach to treatment for angioedema: consesnsus report from the Hereditary Angioedema International Working Group. Allergy 2014;69:602-16. doi: 10.1111/all.12380
  • 2. Zuraw BL. Hereditary angioedema. N Engl J Med 2008;359:1027-36. doi: 10.1056/NEJMcp0803977.
  • 3. Gompels MM, Lock RJ, Abinun M, et al. C1 inhibitor deficiency: consensus document. Clin Exp Immunol 2005;139:379-94.
  • 4. Zilberberg M, Jacobsen T, Tillotson G. The burden of hospitalizations and emergency department visits with hereditary angioedema and angioedema in the United States, 2007. Allergy Asthma Proc 2010;31:511-9. doi: 10.2500/ aap.2010.31.3403.
  • 5. Roche O, Blanch A, Caballero T, Sastre N, Callejo D, Lopez- Trascasa M. Hereditary angioedema due to C1 inhibitor deficiency: patient registry and approach to the prevelance in Spain. Ann Allergy Asthma Immunol 2005;94:498-503.
  • 6. Bygum A. Hereditary angioedema in Denmark: a nationwide survey. Br J Dermatol 2009;161:1153-8. doi: 10.1111/j.1365- 2133.2009.09366.x.
  • 7. Craig T, Aygören-Pürsün E, Bork K, et al. WAO Guideline for the management of hereditary angioedema. World Allergy Organ J 2012;5:182-9. doi: 10.1097/ WOX.0b013e318279affa.
  • 8. Caballero T, Baeza ML, Cabanas R, et al. Consensus statement on the diagnosis, management and the treatment of angioedema mediated by bradikynin. Part II. Treatment, follow up, and special situations. J Investig Allergol Clin Immunol 2011;21:422-41.
  • 9. Costantino G, Casazza G, Bossi I, Duca P, Cicardi M. Longterm prophylaxis in hereditary angio-edema: a systematic review. BMJ Open 2012;2:e000524. doi: 10.1136/ bmjopen-2011-000524.
  • 10. Wilson DA, Bork K, Shea EP, Rentz AM, Blaustein MB, Pullman WE. Economic costs associated with acute attacks and long term management of hereditary angioedema. Ann Allergy Asthma Immunol 2010;104:314-20. doi: 10.1016/j. anai.2010.01.024.
  • 11. Lumry WR, Castaldo AJ, Vernon MK, Blaustein MB, Wilson DA, Horn PT. The humanistic burden of hereditary angioedem: Impact on health-related quality of life, productivity, and depression. Allergy Asthma Proc 2010;31:407-14. doi:10.2500/aap.2010.31.3394.
  • 12. Nordenfelt P, Dawson S, Wahlgren CF, Lindfors A, Malbris L, Björkander J. Quantifying the burden of disease health state in patients with hereditary angiopedema in Sweeden. Allergy Asthma Proc 2014;35:185-90. doi: 10.2500/ aap.2014.35.3738.
  • 13. Cabellero T, Aygören-Pürsün E, Bygum A, et al. The humanistic burden of hereditary angioedema: results from the Burden of Illness Study in Europe. Allergy Asthma Proc 2014;35:47-53. doi: 10.2500/aap.2013.34.3685.
  • 14. Hughes DA, Bagust A, Haycox A, Walley T. Accounting for noncompliance in pharmacoeconomic evaluations. Pharmacoeconomics 2001;19:1185-97.
  • 15. Hudhes DA. Economic impact of poor compliance with pharmaceuticals. Expert Rev Pharmacoecon Outcomes Res 2002;2:327-35. doi: 10.1586/14737167.2.4.327
  • 16. Bygum A, Fagerberg CR, Ponard D, Monnier N, Lunardi J, Drouet C. Mutational spectrum and phenotypesin Danish families with hereditary angioedema because of C1 inhibitor deficiency. Allergy 2011;66:76-84. doi: 10.1111/j.1398- 9995.2010.02456.x
  • 17. Squeglia V, Barbarino A, Bova M, et al. High attack frequency in patients with angioedema due to C1-inhibitor deficiency is a major determinant in switching to home therapy: a real-life observational study. Orphanet J Rare Dis 2016; 11:133.doi: 10.1186/s13023.016.0518-8.
  • 18. Nordenfelt P, Nilsson M, Björkander J, Mallbris L, Lindfors A, Whalgren CF. Hereditary angioedema in Swedish adults: report from the national cohort. Acta Derm Venerol 2016;96:540-5. doi: 10.2340/00015.555.2274.
  • 19. Gomez-Traseira C, Perez-Fernandez E, Lopez-Serrano MC, et al. Clinical pattern and acute and long-term management of hereditary angioedema due to C1-esterase inhibitor deficiency. J Investig Allergol Clin Immunol 2015;25:358- 64.
  • 20. Zanichelli A, Vacchini R, Badini M, Penna V, Cicardi M. Standard care impact on angioedema because of hereditary C1 inhibitor deficiency: a 21-month prospective study in a cohort of 103 patients. Allergy 2011;66:192-6. doi: 10.1111/j.1398-9995.2010.02433.x.
  • 21. Ren HL, Zhang HY. Clinical features of hereditary angioedema: analysis of 133 cases. Zhonghua Yi Xue Za Zhi 2007;87:2772-6.
  • 22. Gelfand JA, Sherins RJ, Alling DW, Frank MM. Treatment of hereditary angioedema with Danazol. Reversal of clinical and biochemical abnormalities. N Engl J Med 1976;295:1444-8. doi: 10.1056/NEJM197.612.232952602.
  • 23. Bork K, Bygum A, Hardt J. Benefits and risks of Danazol in hereditary angioedema: a long term survey of 118 patients. Ann Allergy Asthma Immunol 2008;100:153-61. doi: 10.1016/S1081-1206(10)60424-3.
  • 24. Riedl MA. Critical appraisal of androgen use in hereditary angioedema: a systematic review. Ann Allergy Asthma Immunol 2015;114:281-8. doi: 10.1016/j.anai.2015.01.003.
  • 25. Cicardi M, Suffritti C, Perego F, Caccia S. Novelties in the diagnosis and treatment of angioedema. J Investig Allergol Clin Immunol 2016;26:212-1. doi: 10.18176/jiaci.0087
  • 26. Farkas H, Veszeli N, Csuka D, et al. Management of pregnancies in a hereditary angioedema patients after treatment with attenuated androgens since childhood. J Obstet Gynaecol 2015;35:89-90. doi: 10.3109/01443.615.2014.925860.
  • 27. Zuraw BL, Busse PJ, White M, et al. Nanofiltered C1 inhibitor concentrate for treatment of hereditary angioedema. N Engl J Med 2010;363:513-22. doi: 10.1056/NEJMoa0805538.
  • 28. Craig T, Shapiro R, Vegh A, et al. Efficacy and safety of an intravenous C1-inhibitor concentrate for long-term prophylaxis in hereditary angioedema. Allergy Rhinol (Providence). 2017:1:8:13-19. doi: 10.2500/ar.2017.8.0192
  • 29. Ucar R, Arslan S, Baran M, Caliskaner AZ. Difficulties encountered in the emergency department by patients with hereditary angioedema experiencing acute attacks. Allergy Asthma Proc 2016;37:72-5. doi: 10.2500/aap.2016.37.3905.
  • 30. Kesim B, Uyguner ZO, Gelincik A, et al. The Turkish hereditary angioedema pilot study (TURHAPS): the first Turkish series of hereditary angioedema. Int Arch Allergy Immunol 2011;156:443-50. doi:10.1159/000323915.
  • 31. Zanichelli A, MagerlM, Longhurst H, Fabien V, Maurer M. Hereditary angioedema with C1 inhibitor deficiency: delay indiagnosis in Europe. Allergy Asthma Clin Immunol 2013;9:29. doi: 10.1186/1710-1492-9-29.
  • 32. Frank MM, Gelfand JA, Atkinson JP. Herediatry angioedema: the clinical syndrome and its management. Ann Intern Med 1976;84:580-93.
  • 33. Agostoni A, Aygören-Pürsün E, Binkley KE, et al. Hereditary and acquired angioedema: problems and progress: proceedings of the third C1 esterase inhibitör deficiency workshop and beyond. J Allergy Clin Immunol 2004;114(3 Suppl):S51-131.
  • 34. Agostoni A. Cicardi M. Hereditary and acquired C1-inhibitor deficiency: biological and clinical characteristics in 235 patients. Medicine (Baltimore) 1992;71:206-15.
  • 35. Bork K, Meng G, Staubach P, Hardt J. Hereditary angioedema: new findings concerning symptoms, affected organs, and course. Am J Med 2006;119:267-74.
There are 35 citations in total.

Details

Primary Language English
Subjects Clinical Sciences
Journal Section Articles
Authors

Semra Demır This is me

Derya Unal This is me

Muge Olgac This is me

Asli Gelıncık This is me

Raif Coskun This is me

Bahauddin Colakoglu This is me

Suna Buyukozturk This is me

Publication Date January 29, 2019
Published in Issue Year 2019 Volume: 32 Issue: 1

Cite

APA Demır, S., Unal, D., Olgac, M., Gelıncık, A., et al. (2019). Evaluating adherence to long-term prophylaxis treatment with danazol in adult hereditary angioedema patients: A real life study. Marmara Medical Journal, 32(1), 7-13. https://doi.org/10.5472/marumj.518719
AMA Demır S, Unal D, Olgac M, Gelıncık A, Coskun R, Colakoglu B, Buyukozturk S. Evaluating adherence to long-term prophylaxis treatment with danazol in adult hereditary angioedema patients: A real life study. Marmara Med J. January 2019;32(1):7-13. doi:10.5472/marumj.518719
Chicago Demır, Semra, Derya Unal, Muge Olgac, Asli Gelıncık, Raif Coskun, Bahauddin Colakoglu, and Suna Buyukozturk. “Evaluating Adherence to Long-Term Prophylaxis Treatment With Danazol in Adult Hereditary Angioedema Patients: A Real Life Study”. Marmara Medical Journal 32, no. 1 (January 2019): 7-13. https://doi.org/10.5472/marumj.518719.
EndNote Demır S, Unal D, Olgac M, Gelıncık A, Coskun R, Colakoglu B, Buyukozturk S (January 1, 2019) Evaluating adherence to long-term prophylaxis treatment with danazol in adult hereditary angioedema patients: A real life study. Marmara Medical Journal 32 1 7–13.
IEEE S. Demır, D. Unal, M. Olgac, A. Gelıncık, R. Coskun, B. Colakoglu, and S. Buyukozturk, “Evaluating adherence to long-term prophylaxis treatment with danazol in adult hereditary angioedema patients: A real life study”, Marmara Med J, vol. 32, no. 1, pp. 7–13, 2019, doi: 10.5472/marumj.518719.
ISNAD Demır, Semra et al. “Evaluating Adherence to Long-Term Prophylaxis Treatment With Danazol in Adult Hereditary Angioedema Patients: A Real Life Study”. Marmara Medical Journal 32/1 (January 2019), 7-13. https://doi.org/10.5472/marumj.518719.
JAMA Demır S, Unal D, Olgac M, Gelıncık A, Coskun R, Colakoglu B, Buyukozturk S. Evaluating adherence to long-term prophylaxis treatment with danazol in adult hereditary angioedema patients: A real life study. Marmara Med J. 2019;32:7–13.
MLA Demır, Semra et al. “Evaluating Adherence to Long-Term Prophylaxis Treatment With Danazol in Adult Hereditary Angioedema Patients: A Real Life Study”. Marmara Medical Journal, vol. 32, no. 1, 2019, pp. 7-13, doi:10.5472/marumj.518719.
Vancouver Demır S, Unal D, Olgac M, Gelıncık A, Coskun R, Colakoglu B, Buyukozturk S. Evaluating adherence to long-term prophylaxis treatment with danazol in adult hereditary angioedema patients: A real life study. Marmara Med J. 2019;32(1):7-13.